A Phase 1/2 gene therapy trial conducted at the Children's hospital of Philadelphia have shown promising results in the treatment of haemophillia A. 18 male patients treated with SPK-8011, an AAV based vector encoding Factor VIII. Patients suffering from haemophillia A have a mutation in the gene coding for Factor VIII. This protein can not be produced, which acts as a clotting factor when bleeding occurs. Patients normally exhibit uncontrolled bleeding episodes. Patients treated with this gene therpay vector showed a 91.5% reduction in bleeding episodes.
Multiyear Factor VIII Expression after AAV Gene Transfer for Hemophilia A
DOI: 10.1056/NEJMoa2104205